Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study (ASSURE)

Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study in Patients With Cystic Fibrosis Receiving Enteral Feeding

Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB use, in patients with cystic fibrosis and exocrine pancreatic insufficiency.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Device: RELiZORB (immobilized lipase) cartridge; Other: Impact Peptide 1.5

Detailed Description

Study Entry (Day -14): Baseline blood samples collected for plasma and erythrocyte concentrations of docosahexaenoic acid (DHA) and eicosapentaenoic (EPA). Baseline characteristics collected included BMI and cystic fibrosis related diabetes.

Observation Period (Day -14 to Day -8): Subjects followed their usual enteral nutrition regimen with pancreatic enzyme replacement therapy (PERT).

Run-in Period (Day -7 to Day -1): Subjects used Peptamen 1.5 enteral formula at their normal volume of administration from 500 mL to 1,000 mL per feeding with usual PERT regimen.

Treatment Period (Day 0 to Day 90): Subjects used Impact Peptide 1.5 up to a maximum volume of 1,000 mL per feeding with RELiZORB for the 90 day treatment period. Blood screening measurements were repeated at start of treatment period (Day 0), Day 30, Day 60 and Day 90. PERT use with enteral feedings was prohibited. Safety and tolerability were assessed with GI symptom diaries and systematic assessments of adverse events and unanticipated adverse device effects.

• Study Type: Interventional
• Enrollment (Actual): 49
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Florida
    • Hollywood, Florida, United States, 33021
      • Joe DiMaggio Children's Hospital / Memorial Healthcare System
  • Idaho
    • Boise, Idaho, United States, 83712
      • St. Luke's CF Center of Idaho
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children at Indiana University Health
  • Maine
    • Portland, Maine, United States, 04102
      • Maine Medical Center
  • Michigan
    • Grand Rapids, Michigan, United States, 49503
      • Helen DeVos Children's Hospital CF Care Center
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospital
    • Saint Louis, Missouri, United States, 63104
      • Cardinal Glennon Children's Hospital / Saint Louis University
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
    • Dayton, Ohio, United States, 45404-1815
      • Dayton Children's Hospital
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Children's Hospital of Pittsburgh of UPMC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Confirmed diagnosis of cystic fibrosis
2. Documented history of exocrine pancreatic insufficiency
3. Enteral formula use a minimum of 4x/week, using PERT, consuming an unrestricted fat diet, and willing to use Peptamen 1.5 and Impact Peptide 1.5
4. Written informed consent or assent.

Exclusion Criteria:

1. Uncontrolled diabetes mellitus
2. Signs and symptoms of liver cirrhosis or portal hypertension
3. Lung or liver transplant
4. Active cancer currently receiving cancer treatment
5. Crohn's or celiac disease, infectious gastroenteritis, sprue, lactose intolerance, inflammatory bowel disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Single Arm: Open label RELiZORB Cartridge & Impact Peptide 1.5; RELiZORB (immobilized lipase) cartridge and Impact Peptide 1.5 with enteral feedings ranging from 500 mL to 1,000 mL per feeding for a period of 90 days.	Device: RELiZORB (immobilized lipase) cartridge; Hydrolyzing fats from enteral formula, ex vivo, with in-line enteral feed RELiZORB (immobilized lipase) cartridge; Other: Impact Peptide 1.5; Impact Peptide 1.5 at a volume of administration from 500 mL to 1,000 mL per enteral feeding; Other Names: Enteral Formula

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline of Erythrocyte Omega-3 Index % (DHA+EPA)	Change from baseline Day 0 to Day 90 of erythrocyte tissue composition % of the omega-3 index	Day 0 to Day 90

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Unanticipated Adverse Device Effects (UADE)	A UADE is analogous to a serious adverse event (SAE), defined as an AE, occurring at any exposure to the therapeutic agent, that results in any of the following outcomes: death, life-threatening AE, inpatient hospitalization or prolonged existing hospitalization, a persistent or significant disability or incapacity or a congenital anomaly/birth defect.	RELiZORB Treatment Period (Day 0-Day 90): 90 days with additional 30 days of follow up.
Changes in Plasma Concentration Total DHA+EPA	Changes in plasma concentration total DHA+EPA from baseline (Day 0 to Day 90).	RELiZORB Treatment Period (Day 0-Day 90): 90 days
Erythrocyte Composition (%) of DHA	Changes over time in erythrocyte composition (%) for total DHA in ITT population (n=39)	RELiZORB Treatment Period (Day 0-Day 90): 90 days
Erythrocyte Composition (%) of EPA	Changes over time in erythrocyte composition (%) for EPA in ITT population	RELiZORB Treatment Period (Day 0-Day 90): 90 days
Erythrocyte Composition (%) Ratio of n6/n3 Fatty Acids	Change from baseline to Day 90 in n6/n3 ratio in erythrocytes	RELiZORB Treatment Period (Day 0-Day 90): 90 days
Plasma Composition (%) Ratio of n6/n3 Fatty Acids.	Change over time in n6/n3 ratio in plasma in the ITT population	RELiZORB Treatment Period (Day 0-Day 90): 90 days

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
GI Symptoms	GI symptoms recorded in GI diaries by subject and/or caregiver.	Observation, Baseline and RELiZORB Treatment periods (Day -14 to Day 90): 104 days with additional 30 days of follow up.

Collaborators and Investigators

• Sponsor: Alcresta Therapeutics, Inc.
• Collaborators: Cystic Fibrosis Foundation
• Investigators: Study Director: Madhumalli Sarkar, MD, PhD, Alcresta Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 20, 2016
• Primary Completion (Actual): March 30, 2017
• Study Completion (Actual): March 30, 2017

Study Registration Dates

• First Submitted: April 20, 2016
• First Submitted That Met QC Criteria: April 21, 2016
• First Posted (Estimate): April 25, 2016

Study Record Updates

• Last Update Posted (Actual): August 15, 2018
• Last Update Submitted That Met QC Criteria: July 17, 2018
• Last Verified: June 1, 2018

More Information

Terms related to this study

• Keywords: CF, EPI, diabetes, PERT, LCPUFA, DHA, EPA, Omega-3 index
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: Protocol 0000498-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No